Granted Patent
B2
US 12,331,331 · App. 17/428,344 · Granted Jun 17, 2025
Polynucleotides
Inventors:
Amit Nathwani (Rickmansworth, GB); Jenny McIntosh (Epsom, GB); Romuald Corbau (St Albans, GB); Azadeh Kia (Enfield, GB); Carlos Miranda (Watford, GB)
Assignee:
SPUR THERAPEUTICS LIMITED
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Abstract
The present invention relates to polynucleotides comprising a GBA nucleotide sequence that encodes a GCase protein or fragment thereof and wherein a portion of the coding sequence is not wild type. The present invention further relates to viral particles comprising a recombinant genome comprising the polynucleotide of the invention, compositions comprising the polynucleotides or viral particles, and methods and uses of the polynucleotides, viral particles or compositions.
Claims (7)
1. A non-wild type polynucleotide comprising a beta-glucocerebrosidase (GBA) nucleotide sequence, wherein the GBA nucleotide sequence encodes a β-Glucocerebrosidase (GCase) protein, wherein the GBA nucleotide sequence comprises a sequence that is at least 95% identical to SEQ ID NO: 1, and wherein the GCase protein encoded by the GBA nucleotide sequence has GCase activity and expresses in human liver cells at higher levels compared to a GCase encoded by a wild type GBA nucleotide sequence.
2. The non-wild type polynucleotide of claim 1 , wherein the GBA nucleotide sequence is codon optimized for expression in human liver cells.
3. The non-wild type polynucleotide of claim 1 , wherein the GBA nucleotide sequence comprises a reduced number of CpGs compared to a wild type GBA nucleotide sequence.
4. The non-wild type polynucleotide of claim 1 , wherein the non-wild type polynucleotide further comprises a transcription regulatory element.
5. A viral particle comprising a recombinant genome comprising the non-wild type polynucleotide of claim 1 .
6. A pharmaceutical composition comprising the non-wild type polynucleotide of claim 1 and a pharmaceutically acceptable excipient.
7. A pharmaceutical composition comprising the viral particle of claim 5 and a pharmaceutically acceptable excipient.
Assignments (2)
CHANGE OF NAME
Recorded Nov 6, 2024
From: FREELINE THERAPEUTICS LIMITED
To: SPUR THERAPEUTICS LIMITED
Reel/Frame 069155/0889 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Mar 4, 2022
From: NATHWANI, AMIT; MIRANDA, CARLOS; MCINTOSH, JENNY; KIA, AZADEH; CORBAU, ROMUALD
To: FREELINE THERAPEUTICS LIMITED
Reel/Frame 059175/0969 →
Priority Claims (2)
GB 1901512
· Feb 4, 2019
· national
GB 1917910
· Dec 6, 2019
· national
Continuity (1)
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